Benzinga | Nov 4, 2020 11:50AM EST

Alexion Pharma Highlights Upcoming Data Presentations At American Society Of Hematology Dec. 6, 7

Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that six abstracts have been accepted for presentation at the 62nd Annual Meeting and Exposition of the American Society of Hematology (ASH), taking place virtually from December 5 to 8, 2020. Accepted data include a new analysis from two Phase 3 extension studies that demonstrate similar safety and consistent and durable efficacy of ULTOMIRIS(r) (ravulizumab-cwvz) through 52 weeks when used to treat adults with paroxysmal nocturnal hemoglobinuria (PNH) who are greater than 65 years old compared to those who are 65 years old or younger. New data will also be presented that show the safety and efficacy of ULTOMIRIS when used concomitantly with immunosuppressive therapy (IST) in patients with PNH. A retrospective analysis from the Phase 3 extension study will show the majority of adult patients with PNH and aplastic anemia who received treatment with ULTOMIRIS avoided the need for a transfusion for a period of up to 52 weeks, supporting the use of ULTOMIRIS in PNH patients with or without a history of bone marrow disorder who have not previously received treatment with a complement inhibitor.

In addition, an observational analysis of U.S. claims databases will be presented, highlighting the complexity of identifying patients with atypical hemolytic uremic syndrome (aHUS). The retrospective study shows that prespecified triggers of aHUS were reported in approximately one-third of patients with aHUS, and that long-term clinical disease manifestations persisted in the majority of patients with aHUS who were not treated with SOLIRIS(r) (eculizumab).

The accepted abstracts are listed below and are now available on the ASH website:

ePoster Presentations

Paroxysmal Nocturnal Hemoglobinuria (PNH) Abstracts

Phase 3 Study of Danicopan, an Oral Complement Factor D Inhibitor, As Add-on Therapy to a C5 Inhibitor in Patients with Paroxysmal Nocturnal Hemoglobinuria with Clinically Evident Extravascular Hemolysis. Abstract ID #756 poster presentation, Dec. 5, 2020, 7:00 a.m. -- 3:30 p.m. Pacific Time.

Efficacy and Safety of Concomitant Use of Ravulizumab and IST in Patients with Paroxysmal Nocturnal Hemoglobinuria up to 52 Weeks. Abstract ID #1686 -- poster presentation, Dec. 6, 2020, 7:00 a.m. -- 3:30 p.m. Pacific Time.

Risk Factors for Thrombotic Events in Patients with PNH: A Nested Case-Control Study in the International PNH Registry. Abstract ID #2457-- poster presentation, Dec. 6, 2020, 7:00 a.m. -- 3:30 p.m. Pacific Time.

Efficacy and Safety of Ravulizumab in Older Patients Aged >65 years With Paroxysmal Nocturnal Hemoglobinuria in the 301 and 302 Phase 3 Extension studies. Abstract ID #2586 -- poster presentation, Dec. 7, 2020, 7:00 a.m. -- 3:30 p.m. Pacific Time.

Transfusion Requirements in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria with or without a History of Bone Marrow Disorder Receiving Ravulizumab and Eculizumab: Results from a Phase 3 Non-Inferiority Study Extension. Abstract ID #2575 -- poster presentation, Dec. 7, 2020, 7:00 a.m. -- 3:30 p.m. Pacific Time.

Atypical Hemolytic Uremic Syndrome (aHUS) Abstracts

Triggers in Patients with Atypical Hemolytic Uremic Syndrome: An Observational Cohort Study Using a U.S. Claims Database. Abstract ID #1579-- poster presentation, Dec. 5, 2020, 7:00 a.m. -- 3:30 p.m. Pacific Time.